Phenotypic cure 6 years after bone marrow transplantation in a β-thalassemia patient

Sivan Berger-Achituv, Michal Rothschild, Elizabeth Naparstek, Baruch Wolach

Research output: Contribution to journalArticlepeer-review

Abstract

A β-thalassemia major (TM) male patient underwent bone marrow transplantation from a fully matched carrier sister at age three. He continued to be transfusion dependent although chimerism evaluation, using analysis of the β-globin gene mutation, showed 90-98% donor cells in peripheral leukocytes after 3 months and 82-90% donor cells in the bone marrow after 26 and 30 months. Clinical improvement occurred gradually, and the patient became transfusion free 6 years posttransplant. We reason that an exceptional slow displacement of host red-blood-cell lineage took place that was not evident from the high level of chimerism found in peripheral leukocytes and bone marrow.

Original languageEnglish
Pages (from-to)373-375
Number of pages3
JournalAmerican Journal of Hematology
Volume84
Issue number6
DOIs
StatePublished - Jun 2009

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